Gene therapy for gaucher disease: 5-Year safety check begins
NCT ID NCT06545136
First seen Nov 05, 2025 · Last updated May 22, 2026 · Updated 25 times
Summary
This study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will track side effects and how long the treatment's effects last over 5 years. The goal is to understand the long-term safety and durability of this one-time gene therapy.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Hospital Quironsalud Zaragoza
Zaragoza, Spain
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Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
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Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
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Salford Royal Hospital
Salford, United Kingdom
Conditions
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